Actively Recruiting
Maralixibat in Patients With Cystic Fibrosis and Constipation
Led by Children's Hospital Los Angeles · Updated on 2026-04-21
20
Participants Needed
1
Research Sites
116 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Standard osmotic laxatives often provide inadequate relief in this population. Maralixibat is an ileal bile acid transporter inhibitor (IBATi) that increases the amount of bile acids reaching the colon. Bile acids can enhance intestinal secretion, reduce transit time, and soften stool. This study will evaluate whether Maralixibat improves stool consistency in children with CF who experience constipation. We will enroll 20 children with CF and constipation, defined as a Bristol Stool Scale score \<4 for at least one week while on a stable laxative regimen. Each participant will receive Maralixibat for two weeks in addition to their usual laxatives. Families will record stool consistency and ease of defecation before and during treatment. The primary objective is to determine whether Maralixibat improves stool consistency to a Bristol Stool Scale score \>4. The secondary objective is to assess changes in ease of defecation using standardized questionnaires.
CONDITIONS
Official Title
Maralixibat in Patients With Cystic Fibrosis and Constipation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 1 to 18 years
- Confirmed diagnosis of cystic fibrosis by genetic testing or sweat chloride test
- Chronic constipation defined as Bristol Stool Scale score less than 3 while on stable constipation treatment
- Stable constipation medication regimen with no changes or dose adjustments for at least 4 weeks prior to enrollment
- Conventional therapy may include stool softeners, stimulant laxatives, or dietary interventions
You will not qualify if you...
- Uncontrolled deficiency of fat-soluble vitamins A, D, E, or K
- Changes to constipation medication regimen within 4 weeks prior to starting Maralixibat
- Adequately treated constipation with Bristol Stool Scale score greater than 3 on current regimen
- Known allergy or sensitivity to Maralixibat or any study ingredients
- Inability or unwillingness to provide written informed consent by participant or guardian
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
Research Team
J
Jaya Punati, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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